학술논문
The Arduous Path to Drug Approval for the Management of Prader–Willi Syndrome: A Historical Perspective and Call to Action.
Document Type
Article
Author
Source
Subject
*PRADER-Willi syndrome
*DRUG approval
*MEDICAL genetics
*HUMAN growth hormone
*SELF-injurious behavior
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Language
ISSN
1661-6596
Abstract
Despite the well-established burden of illness and the social costs of PWS, the only medication that has been approved by the Food and Drug Administration (FDA) for the management of this disorder is recombinant human growth hormone (rhGH), which was approved in 2000 [[7]]. The Arduous Path to Drug Approval for the Management of Prader-Willi Syndrome: A Historical Perspective and Call to Action Prader-Willi syndrome (PWS) is a neuroendocrine genetic disorder resulting from the loss of paternally expressed imprinted genes in chromosome 15q11-q13 [[1]]. [Extracted from the article]